Multiple myeloma is a blood cancer that affects the bone marrow, where there are white blood cells, plasma cells, which are responsible for producing the antibodies needed to fight infections. In multiple myeloma, these cells undergo an abnormal growth process and form tumors in some areas of the bones. Although there are several treatments for this disease, which is the second most common blood cancer, there are patients who do not respond and have a very limited life expectancy.
Hospital Clínic has developed the first CAR-T in Europe for these people without a therapeutic alternative, achieving complete remission of the disease in 18 of them in the first clinical trial of this new treatment. CAR-T is a type of cell and gene therapy that turns the patient into their own donor. It consists of modifying lymphocytes so that they have the ability to attack tumor cells. Through apheresis, a technique that allows the separation of blood components, T lymphocytes, responsible for the immune response, are obtained. These lymphocytes are genetically reprogrammed so that they can recognize and attack tumor cells, once they have been transfused back into the patient.
It is not the first time that the Barcelona center has created a therapy of this type, which arise from the ARI project. This initiative was born with Ari Benedé, who was diagnosed with leukemia when she was 13 years old. Her family began to search for possible treatments around the world and found that CAR-T existed in the United States. It was a very expensive treatment and Ari and her family began to raise funds to be able to bring it to Barcelona. Ari died in 2016, but her efforts were the origin of several therapies that Hospital Clínic has promoted in recent years and that are beginning to show results.
The first was CAR-T ARI-0001, for acute lymphoblastic leukemia (ALL), the disease Ari had. In this case, the Spanish Agency of Medicines and Medical Devices (AEMPS) approved its use as a medicine at the beginning of the year, becoming the first CAR-T developed entirely in Europe that has been approved by a regulatory agency and that, furthermore, is public, with a more affordable cost for the healthcare system.
The second CAR-T from Clínic, the one for multiple myeloma, known as ARI-0002h, is preparing the documentation to request approval from the AEMPS for its use as a medicine, after obtaining good results in the clinical trial, carried out in collaboration with the Clínica Universidad de Navarra, the Hospital Universitario de Salamanca, the Hospital Virgen de la Arrixaca de Murcia and the Hospital Virgen del Rocío de Sevilla and with the support of the La Caixa Foundation.
Specifically, the trial, with 30 patients, has shown that all improve with this treatment and 60% have a complete remission of the disease. It is expected that the new CAR-T can be approved by the AEMPS by the summer of 2022. After these first two gene therapies, the Barcelona center is already working on new ones, still in an initial phase.